MX2020009487A - Systems and methods for the treatment of hemoglobinopathies. - Google Patents

Systems and methods for the treatment of hemoglobinopathies.

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Publication number
MX2020009487A
MX2020009487A MX2020009487A MX2020009487A MX2020009487A MX 2020009487 A MX2020009487 A MX 2020009487A MX 2020009487 A MX2020009487 A MX 2020009487A MX 2020009487 A MX2020009487 A MX 2020009487A MX 2020009487 A MX2020009487 A MX 2020009487A
Authority
MX
Mexico
Prior art keywords
methods
systems
hemoglobinopathies
treatment
portions
Prior art date
Application number
MX2020009487A
Other languages
Spanish (es)
Inventor
Jennifer Leah Gori
De Lamothe-Dreuzy Edouard Aupepin
Jack Heath
John Anthony Zuris
Kaihsin Chang
Original Assignee
Editas Medicine Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Editas Medicine Inc filed Critical Editas Medicine Inc
Publication of MX2020009487A publication Critical patent/MX2020009487A/en

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    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/12—Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/14—Blood; Artificial blood
    • A61K35/17—Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/20—Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Chemical & Material Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
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  • Microbiology (AREA)
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  • Medicinal Chemistry (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Cell Biology (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Virology (AREA)
  • Developmental Biology & Embryology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Diabetes (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)

Abstract

Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the <i>HBG1 </i>and <i>HBG2</i> loci, portions of the erythroid specific enhancer of the <i>BCL11A</i> gene, or a combination thereof, in cells and increasing expression of fetal hemoglobin.
MX2020009487A 2018-03-14 2019-03-14 Systems and methods for the treatment of hemoglobinopathies. MX2020009487A (en)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201862643168P 2018-03-14 2018-03-14
US201862767488P 2018-11-14 2018-11-14
US201862773073P 2018-11-29 2018-11-29
PCT/US2019/022374 WO2019178426A1 (en) 2018-03-14 2019-03-14 Systems and methods for the treatment of hemoglobinopathies

Publications (1)

Publication Number Publication Date
MX2020009487A true MX2020009487A (en) 2020-12-10

Family

ID=65952168

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2020009487A MX2020009487A (en) 2018-03-14 2019-03-14 Systems and methods for the treatment of hemoglobinopathies.

Country Status (12)

Country Link
US (1) US20210254061A1 (en)
EP (1) EP3765614A1 (en)
JP (1) JP2021518102A (en)
KR (1) KR20200132924A (en)
CN (1) CN112020558A (en)
AU (1) AU2019236209A1 (en)
BR (1) BR112020018364A2 (en)
CA (1) CA3093702A1 (en)
IL (1) IL277234A (en)
MX (1) MX2020009487A (en)
SG (1) SG11202008956XA (en)
WO (1) WO2019178426A1 (en)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3981876A1 (en) 2014-03-26 2022-04-13 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating sickle cell disease
IL260257B2 (en) * 2015-12-28 2024-05-01 Novartis Ag Compositions and methods for the treatment of hemoglobinopathies
EP3596217A1 (en) 2017-03-14 2020-01-22 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
EP3622070A2 (en) 2017-05-10 2020-03-18 Editas Medicine, Inc. Crispr/rna-guided nuclease systems and methods
EP3635120A1 (en) * 2017-06-02 2020-04-15 Institut National de la Santé et de la Recherche Médicale (INSERM) Recombinant lentiviral vector for stem cell-based gene therapy of sickle cell disorder
EP3652312A1 (en) 2017-07-14 2020-05-20 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
WO2020033774A1 (en) * 2018-08-08 2020-02-13 Integrated Dna Technologies, Inc. Novel mutations that enhance the dna cleavage activity of acidaminococcus sp. cpf1
US20220047637A1 (en) * 2018-11-29 2022-02-17 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
IL293643A (en) * 2019-12-08 2022-08-01 Editas Medicine Inc Modified cells and methods for the treatment of hemoglobinopathies
AU2021218811A1 (en) * 2020-02-13 2022-09-01 Beam Therapeutics Inc. Compositions and methods for engraftment of base edited cells
US20230279438A1 (en) * 2020-05-13 2023-09-07 Inserm (Institut National De La Santé Et La Recherche Médicale) Base editing approaches for the treatment of betahemoglobinopathies
AU2022220203A1 (en) * 2021-02-11 2023-08-24 Koninklijke Nederlandse Akademie Van Wetenschappen Curing disease by transcription regulatory gene editing
EP4047095A1 (en) * 2021-02-22 2022-08-24 Eberhard Karls Universität Tübingen Medizinische Fakultät Method and composition for a targeted gene knockout
WO2023014727A1 (en) * 2021-08-02 2023-02-09 Editas Medicine, Inc. Systems and methods for the treatment of hemoglobinopathies
CN115851706A (en) * 2021-09-24 2023-03-28 华东师范大学 Base editing system and application thereof
KR20230132341A (en) 2022-03-07 2023-09-15 광주과학기술원 Cpf1 mutant protein for rapid DNA diagnosis

Family Cites Families (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102380245B1 (en) 2013-11-07 2022-03-30 에디타스 메디신, 인코포레이티드 CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS
EP3553176A1 (en) 2014-03-10 2019-10-16 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating leber's congenital amaurosis 10 (lca10)
WO2015148860A1 (en) 2014-03-26 2015-10-01 Editas Medicine, Inc. Crispr/cas-related methods and compositions for treating beta-thalassemia
WO2016073990A2 (en) 2014-11-07 2016-05-12 Editas Medicine, Inc. Methods for improving crispr/cas-mediated genome-editing
US10900034B2 (en) * 2014-12-03 2021-01-26 Agilent Technologies, Inc. Guide RNA with chemical modifications
WO2016094872A1 (en) * 2014-12-12 2016-06-16 The Broad Institute Inc. Dead guides for crispr transcription factors
CA2986310A1 (en) 2015-05-11 2016-11-17 Editas Medicine, Inc. Optimized crispr/cas9 systems and methods for gene editing in stem cells
US20180273609A1 (en) * 2015-11-04 2018-09-27 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
US20190233814A1 (en) * 2015-12-18 2019-08-01 The Broad Institute, Inc. Novel crispr enzymes and systems
CN109153994A (en) * 2016-03-14 2019-01-04 爱迪塔斯医药公司 For treating β-hemoglobinopathy CRISPR/CAS correlation technique and composition
KR102670601B1 (en) 2016-04-19 2024-05-29 더 브로드 인스티튜트, 인코퍼레이티드 The novel CRISPR enzyme and system
WO2017191503A1 (en) * 2016-05-05 2017-11-09 Crispr Therapeutics Ag Materials and methods for treatment of hemoglobinopathies
EP3487523B1 (en) * 2016-07-19 2023-09-06 Duke University Therapeutic applications of cpf1-based genome editing
MX2020003339A (en) * 2017-10-02 2020-11-06 Genedit Inc Modified cpf1 guide rna.
AU2018383712A1 (en) * 2017-12-11 2020-07-02 Editas Medicine, Inc. Cpf1-related methods and compositions for gene editing
US20190241911A1 (en) * 2018-02-06 2019-08-08 Ohio State Innovation Foundation Engineered guide rna and uses thereof

Also Published As

Publication number Publication date
IL277234A (en) 2020-10-29
WO2019178426A1 (en) 2019-09-19
EP3765614A1 (en) 2021-01-20
AU2019236209A1 (en) 2020-10-01
US20210254061A1 (en) 2021-08-19
KR20200132924A (en) 2020-11-25
CN112020558A (en) 2020-12-01
JP2021518102A (en) 2021-08-02
SG11202008956XA (en) 2020-10-29
CA3093702A1 (en) 2019-09-19
BR112020018364A2 (en) 2020-12-29

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